We are committed to developing innovative and accessible therapies to strengthen the lives of people living with rare blood disorders. Sickle cell disease and beta-thalassemia affect millions of people worldwide, and the impact of these diseases on the health and lives of patients and their families motivates our work each and every day.
Sickle cell disease
Sickle cell disease (SCD) is the most common type of inherited hemoglobinopathy and is driven by structural abnormalities in red blood cells (RBCs). SCD is characterized by debilitating pain, progressive multi-organ damage, and early death. Beginning early in life, people with SCD suffer from blocked blood flow to tissues, known as vaso-occlusion, destruction of RBCs, known as hemolysis, and inadequate oxygen delivery, or hypoxia. The most common complication of SCD is pain, often a consequence of vaso-occlusive crises (VOCs). A VOC occurs when circulation is obstructed by sickled RBCs, causing tissue damage to the organ and resultant pain.
The outcomes of these events begin presenting early in childhood and quickly lead to heart and lung complications; renal disfunction; priapism (prolonged refractory penile erection); spleen enlargement and failure; stroke, retinopathy and mental and physical disabilities. Acute chest syndrome (ACS) occurs in approximately half of all people with SCD and is a leading cause of hospitalization and death from SCD. SCD patients on average have a significantly shorter lifespan than healthy adults.
The global incidence of SCD is estimated to be approximately 300,000 births annually. In the United States, where newborn screening for SCD is mandatory, the estimated prevalence is approximately 100,000 individuals. In the European Union, the estimated prevalence is approximately 134,000 individuals. The global prevalence of SCD is estimated to be approximately 4.4 million patients. SCD is most common among people of African, Middle Eastern and South Asian descent.
Beta-thalassemia, which is part of the second group of hemoglobinopathies, is a rare inherited red blood cell disorder. Beta-thalassemia presents as a spectrum of disease, with patients categorized based on hemoglobin levels and clinical manifestations. If left untreated, the disease causes severe anemia, splenomegaly, skeletal abnormalities, organ failure and early death.
The prevalence of beta-thalassemia globally is estimated to be 288,000, with an incidence of 60,000 births per year, and it is especially prevalent in developing countries of Africa, South Asia, Southeast Asia, the Mediterranean region and the Middle East. The total combined prevalence of beta thalassemia in the European Union and United States is estimated to be approximately 19,000 patients.
These groups can provide information on rare diseases in the U.S., including sickle cell disease and beta thalassemia.
National Heart Lung Blood Institute: http://www.nhlbi.nih.gov
Office of Rare Diseases: https://rarediseases.info.nih.gov
Global Genes: https://globalgenes.org
FDA Office of Minority Health: http://www.fda.gov/AboutFDA/CentersOffices/OC/OfficeofMinorityHealth/default.htm
National Organization for Rare Disorders (NORD): http://rarediseases.org
These groups can help connect patients to information, resources and support.
Foundation for Sickle Cell Disease Research: https://fscdr.org/
Sickle Cell Disease Association of America: http://www.sicklecelldisease.org
American Sickle Cell Anemia Association: http://www.ascaa.org
Sickle Cell Warriors: http://sicklecellwarriors.com
Sickle Cell Society: http://sicklecellsociety.org
Thalassemia Support Foundation: http://www.helpthals.org
Cooley’s Anemia Foundation https://www.thalassemia.org/about-the-foundation/our-mission/
Imara Expanded Access Policy
Imara is dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies. Sickle cell disease represents a critical unmet medical need globally in which it is considered as a rare disease in many parts of the world, including in the United States, and as an endemic condition in several African countries. Imara’s lead product candidate, IMR-687, is under development for the treatment of sickle cell disease. Currently, Imara does not have an expanded access program for IMR-687.
Expanded access may provide an avenue to use an investigational product, such as IMR-687, outside a clinical trial to diagnose, monitor, or treat a serious condition or disease in a patient. Imara has used guidelines from the US Food and Drug Administration (US FDA) and other regulatory agencies to develop the following criteria for when expanded access may be made available on a case-by-case basis for individual patients:
- Ongoing or planned clinical studies are not available to the patient, including lack of access due to geographical location of potential clinical trial sites,
- Significant evidence exists that supports both the safety and the efficacy of the investigational drug for the indication,
- The potential benefits to the particular patient seeking access to the investigational drug outweigh the potential risks to the patient,
- An adequate supply of the investigational drug exists,
- All necessary regulatory/institutional approvals have been obtained to allow drug administration, and
- The request for expanded access has been made by a qualified health care provider with expertise appropriate for the administration of the drug and for monitoring and managing the patient.
Imara supports expanded access programs and the need for a suitable policy, and it intends to provide patients with sickle cell disease access to IMR-687 at a suitable time and in the correct method when used outside a clinical trial. At this time, Imara believes that the most appropriate way to use our investigational lead product candidate, IMR-687, is by participation in one of our clinical trials (https://clinicaltrials.gov/). If you have additional questions, please speak with your health care provider or contact firstname.lastname@example.org. Imara will typically respond to requests sent to this email address within five business days.
Imara may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov after such record becomes active.